Scleroderma, also referred to as
systemic sclerosis, is a disease of the connective tissue. Scleroderma is
characterized by the formation of scar tissue (fibrosis) in the skin and organs
of the body. The cause of scleroderma is not known. Researchers have found some
evidence that genes are important factors. This means that inheritance at least
plays a partial role. It is not unusual to find other autoimmune diseases in the
families of scleroderma patients. The disease is more frequent in females than
in males.

Below are perspectives on key reports presented
at the recent national meeting of the American College of Rheumatology:

Dr. Shiel's Perspective: Recognizing that lung scarring conditions can
seriously damage the lung, and that Cytoxan can improve many lung scarring
conditions, it is now difficult to recommend this potentially toxic drug for the
scarring form of scleroderma lung (pulmonary fibrosis). This form of lung
disease must be distinguished from inflammation of the lungs' tiny air sacs
(alveolitis), as in the next report below.

Inflammation of the lungs' tiny air sacs
(alveolitis) stabilized or improved in scleroderma patients treated with
cyclophosphamide (Cytoxan) and prednisone.

Dr. Shiel's Perspective: This form of lung disease (as compared to the
one described in the report above) actually did respond to Cytoxan. The key
element seems to be inflammation. If inflammation is present in the lungs, it
may be helped by Cytoxan treatments (typically given as a monthly intravenous
infusion). The exact form of lung disease requires a lung biopsy for a precise
diagnosis.

Specific genes were found responsible for why
some scleroderma patients develop lung disease while others do not.

Dr. Shiel's Perspective: This has always been a perplexing phenomenon
with autoimmune rheumatic diseases - why they present so differently from
patient to patient. It may be that environmental differences account for some of
this, but it seems that there are also subtle differences genetically in the
inherited material of each patient that may be playing a significant role.

Dr. Shiel's Perspective: Because Viagra has an effect on the smooth
muscle of the blood vessels, it was tried in these very ill patients who had
failed the traditional prostacyclin intravenous infusions. Although Viagra did
show favorable results, these are very small preliminary studies. Incidentally,
it might be expected that there would be some benefit on the Raynaud's
phenomenon in patients treated with this drug, and that is exactly what the
researchers saw in all patients. Further research studies are now needed to
really determine the exact role and safety of Viagra in patients with
scleroderma.

L-carnitine seemed to help Raynaud's
and ulcers in scleroderma patients as compared to a placebo.

Dr. Shiel's Perspective: This study was designed to also determine if
L-carnitine would help the skin disease and overall patient function, which it
did not. According to the researchers, more studies are necessary to confirm
whether or not L-carnitine really has benefits. I agree.

Dr. Shiel's Perspective: Again, this was a preliminary study. These
studies, even when unsuccessful, are very critical in establishing which are the
best candidate treatments for long-term research trials. Tamoxifen is out.

Dr. Shiel's Perspective: Stomach and bowel muscles can become weakened as
a result of scleroderma, thereby resulting in loss of normal function of the
bowels. It is a serious condition. Doctors have used bowel muscle-stimulating
drugs, such as metoclopramide, in the past. Doctors have also used antibiotics
(I use tetracycline or erythromycin). The reason for the antibiotics is that the
bowel of scleroderma patients can become overgrown with bacteria, which is
associated with slowing the bowel function. The antibiotics seem to improve this
condition. This study is a head-to-head comparison of the two options. It is
encouraging to see that common antibiotics can be beneficial in this condition.

The outcome of 19 scleroderma
patients receiving stem cell transplantation at various university centers was
reported.

Dr. Shiel's Perspective: Some of the studied patients had progressive
scleroderma disease. 2 of the subjects died. Some stabilized their disease. A
few had improvement of their skin and function. Stem cell transplantation is
known to be extremely dangerous and requires powerful doses of
immune-suppressing drugs. This study argues for more studies that could
distinguish which patients might really benefit from this procedure.